Emugen MECP2 gene therapy

Emugen Therapeutics is developing an adeno-associated virus (AAV) gene therapy for the treatment of Rett Syndrome, a rare neurodevelopmental disorder caused by mutations in the *MECP2* gene. The therapy utilizes Emugen's proprietary platform designed to address haploinsufficiency by targeting "poison exons"—highly conserved, non-coding sequences that naturally regulate gene expression through alternative splicing. By preventing the inclusion of these poison exons, the therapy aims to restore functional Methyl-CpG-binding protein 2 (MECP2) levels to therapeutic ranges. The program is currently in the discovery and proof-of-concept stage, focusing on precision restoration of gene expression in the central nervous system.

Other names

Emugen Rett Syndrome Program